Prophylaxis use of clotting factor replacement products in people with non-severe haemophilia: A review of the literature

Alfonso Iorio, Christoph Königs, Mark T. Reding, Dawn Rotellini, Mark W. Skinner, Maria Elisa Mancuso, Erik Berntorp

Research output: Contribution to journalReview articlepeer-review


Introduction: People with non-severe haemophilia appear to be under-treated in many countries, and this may lead to joint damage and worsen quality of life. Aim: To review literature for clotting factor replacement prophylaxis in people with non-severe haemophilia A and B (HA/HB) in relation to long-term outcomes to support clinical decision-making. Methods: A targeted literature search was performed to identify studies published between 2000 and 2021 that included prophylaxis in people with non-severe HA/HB and long-term outcomes, including annualized bleeding rates, joint health and quality of life. Results: Although eligible articles included 2737 and 2272 people with mild or moderate HA, respectively, only 22% (n = 609) and 29% (n = 668) reported treatment regimens. A total of 549 people with moderate HA were treated with factor replacement prophylaxis and were from high-income countries. On the contrary, nearly all people with mild HA received desmopressin (n = 599). Details of treatment regimens for women with haemophilia and people with HB were sparse. Three studies provided long-term outcomes for people with moderate haemophilia who received prophylaxis with factor concentrate, supporting early prophylaxis in people with a frequent bleeding phenotype regardless of their endogenous clotting factor level to preserve joint health. Conclusion: There remain large knowledge gaps when considering how to provide optimal treatment for people with non-severe haemophilia. Nonetheless, there is a strong rationale that prophylaxis should be considered early in life according to similar strategies as for severe haemophilia for those with a frequent severe bleeding phenotype.

Original languageEnglish
Pages (from-to)33-44
Issue number1
Publication statusPublished - 2023

Subject classification (UKÄ)

  • Hematology

Free keywords

  • arthropathy
  • bleeding
  • episodic/on-demand treatment
  • non-severe (mild or moderate) haemophilia A or B
  • prophylactic FVIII or FIX
  • women with haemophilia (symptomatic carriers)


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